Agios Pharmaceuticals (NASDAQ:AGIO) announced today that the U.S. Food and Drug Administration (FDA) has approved AQVESME™ (mitapivat) for the treatment of anemia in adults with alpha- or beta-thalassemia.
The decision makes AQVESME the first oral, disease-modifying medicine authorized for the full spectrum of the disease, covering both non-transfusion-dependent (NTD) and transfusion-dependent (TD) patients.
The approval fills a critical gap for approximately 6,000 adult patients in the U.S. living with thalassemia, a rare blood disorder that causes the body to produce an abnormal form of hemoglobin.
While the drug is already marketed as PYRUKYND® for pyruvate kinase (PK) deficiency, Agios will launch the thalassemia indication under the brand name AQVESME in late January 2026.
This rebranding is tied to a specific Risk Evaluation and Mitigation Strategy (REMS) mandated by the FDA to manage potential liver safety risks.
The FDA's authorization was based on the Phase 3 ENERGIZE and ENERGIZE-T trials, which collectively enrolled 452 patients.
The studies demonstrated that by activating the pyruvate kinase enzyme, the drug increases ATP production, which in turn improves red blood cell health and survival.
In the ENERGIZE trial (NTD patients), 42.3% of those treated achieved a significant hemoglobin response compared to 1.6% in the placebo group.
Patients also reported a clinically meaningful reduction in fatigue.
In the ENERGIZE-T trial (TD patients), 30.4% of patients achieved a 50% or greater reduction in transfusion burden.
Notably, 10% of treated patients achieved total transfusion independence for at least eight consecutive weeks.
The path to approval was marked by a regulatory extension to finalize the AQVESME REMS program.
During clinical trials, five patients experienced adverse reactions suggestive of hepatocellular (liver) injury, with two requiring hospitalization.
In all cases, liver tests improved after the drug was discontinued.
Under the REMS program, healthcare providers must conduct liver function tests prior to the first dose, every four weeks for the first 24 weeks of treatment, and as clinically indicated thereafter.
The company confirmed that common side effects observed were generally manageable, including headache and insomnia.
With AQVESME set for a late January launch, Agios shifts its focus to its next major milestone: a Phase 3 readout for mitapivat in Sickle Cell Disease, expected in 2026.
Success in that larger population would solidify the company’s "PK-activator" platform as a foundational pillar of modern hematology.